Zolgensma (onasemnogene abeparvovec)
Zolgensma (onasemnogene abeparvovec) is a medication used to treat children less than 2 years old with spinal muscular atrophy (SMA).
It is given as a one-time-only dose.
- Disease Indications : Spinal Muscular Atrophy
- Manufacturer : AveXis, Inc.
- Usage : Intravenous
Medicine approved by
- European Medical Agency (EMA)
- Food and Drug Administration (FDA)
- Health Canada
Details
What is Zolgensma (onasemnogene abeparvovec) for?
Zolgensma (onasemnogene abeparvovec) is an adeno-associated virus vector-based gene therapy indicated for the treatment of:
- Children less than 2 years old with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or
- Children less than 2 years old with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.
It is given as a one-time-only dose.
Zolgensma (onasemnogene abeparvovec) is available in vial form containing 2 × 10^13 vector genomes/mL solution for infusion. It is provided as a customized kit to meet dosing requirements for each patient and comes with vials of onasemnogene abeparvovec.
How does Zolgensma (onasemnogene abeparvovec) work?
Onasemnogene abeparvovec, the active ingredient in Zolgensma, works by targeting the underlying mechanism of SMA: a lack in the survival of motor neuron (SMN) protein. This protein is crucial for the function of nerves that control muscle movements. In SMA patients, nerve cells are unable to work properly, and over time this leads to muscle weakness.
In most cases of SMA, the SMN1 gene is mutated. The production of SMN protein is directed primarily by SMN1, and partly by SMN2. However, the protein produced by the SMN2 gene is not stable.
Onasemnogene abeparvovec is a vector that contains a functional copy of the SMN gene. When injected, it passes into the nerves from where it replaces the function of the missing or nonworking SMN1 gene and it will tell the motor neuron cells to start making SMN protein. The new gene does not become part of the child’s DNA.
Onasemnogene abeparvovec is an adeno-associated virus vector-based gene therapy. This type of virus does not make people sick.
Although Zolgensma (onasemnogene abeparvovec) keeps motor neuron cells functioning and keeping strength in the muscles, it is not a cure and cannot reverse the damage already caused by SMA before treatment.
Where has Zolgensma (onasemnogene abeparvovec) been approved?
Zolgensma (onasemnogene abeparvovec) was approved for the treatment of children aged 2 years and below with SMA by:
- Food and Drug Administration (FDA), USA on May 24, 2019.
- European Medicines Agency (EMA) on May 18, 2020.
- Health Canada on December 16, 2020.
Zolgensma (onasemnogene abeparvovec) received Fast Track, Breakthrough Therapy, and Priority Review designations by the FDA. Additionally, it received Orphan Drug designation, which provides incentives to encourage the development of drugs for rare diseases.
The FDA also awarded the manufacturer a rare pediatric disease priority review voucher, under a program intended to encourage the development of new drugs and biological products for the prevention and treatment of certain rare pediatric diseases.
Please note that this medicine may have also been approved in other regions than the ones we’ve listed. If you have a question about its approval in a specific country feel free to contact our support team.
How is Zolgensma (onasemnogene abeparvovec) taken?
The standard dosage is:
- A one-time-only dose based on the child’s body weight. Dosage is between 3.3 x 10^14 vector genomes (vg)/kg body weight and 2.31 x 10^15 vg/kg.
The medicine is given via slow intravenous infusion (into a vein) over 60 minutes.
Before Zolgensma (onasemnogene abeparvovec) can be given, several lab tests need to be completed:
- A diagnosis of SMA has to be confirmed
- An adeno-associated virus 9 (AAV9) antibody test to measure the amount of anti-AAV9 antibodies in your child’s blood
- Blood tests to check your child’s liver function and to determine baseline levels of platelet and troponin-I in the blood
A course of an oral corticosteroid, to be determined by the treating doctor, should be started the day before infusion with Zolgensma (onasemnogene abeparvovec). On the day of infusion, the second dose of the oral corticosteroid should be given. This helps manage increased liver enzyme reactions to the medicine by the body’s immune system.
Warning: Viral respiratory infection before or after infusion of the medicine can lead to more serious complications. If you see signs of a possible viral respiratory infection such as coughing, wheezing, sneezing, runny nose, sore throat, or fever, contact the treating doctor immediately.
Complete information about Zolgensma (onasemnogene abeparvovec) dosage (modification) and administration can be found in the official prescribing information listed in our references section.
Note: Please consult with your treating doctor for personalised dosing.
Are there any known adverse reactions or side effects of Zolgensma (onasemnogene abeparvovec)?
Common adverse reactions
The most common adverse reactions (≥10% of patients) listed in the prescribing information include:
- Increased liver enzymes
- Vomiting
Use in a specific population
- Renal (kidney) impairment
- Hepatic (liver) impairment
- 0SMN1/1SMN2 genotype
- Anti-AAV9 antibodies
- Paediatric population
The safety and efficacy of onasemnogene abeparvovec have not been determined in patients with kidney dysfunction. Onasemnogene abeparvovec therapy should be carefully considered. A dose adjustment should not be considered.
Onasemnogene abeparvovec has not been studied in patients with liver dysfunction. The medicine should not be infused unless increased bilirubin is associated with neonatal jaundice. Onasemnogene abeparvovec therapy should be carefully considered in patients with liver dysfunction. A dose adjustment should not be considered.
No dose adjustment should be considered in patients with a bi-allelic mutation of the SMN1 gene and only one copy of SMN2 (see official prescribing information).
No dose adjustment should be considered in patients with baseline anti-AAV9 antibody titres above 1:50 (see official prescribing information).
The safety and efficacy of Zolgensma (onasemnogene abeparvovec) in premature neonates before reaching full term gestational age are not known. Treatment with this medicine should be carefully considered because concomitant treatment with corticosteroids may have negative effects on neurological development.
There is little data on the safety and efficacy of Zolgensma (onasemnogene abeparvovec) in patients 2 years of age and older or with body weight above 13.5 kg. A dose adjustment should not be considered (see official prescribing information).
If you want to explore whether Zolgensma (onasemnogene abeparvovec) is right for your child, discuss the benefits and risks of treatment with your child’s doctor.
For a comprehensive list of side effects and adverse reactions please refer to the official prescribing information.
References
- Full prescribing information [FDA]: Zolgensma (onasemnogene abeparvovec) [PDF] AveXis, May 2019
- What is Spinal Muscular Atrophy? SMA News Today article, cited Mar 2021
- What is Zolgensma? Zolgensma.com, cited Mar 2021
- FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality FDA press release, May 2019
- Zolgensma EMA product page, cited Mar 2021
- Health Canada approves Zolgensma®, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA) Health Canada press release, Dec 2020
Clinical trials
The Food and Drug Administration (FDA), USA approval of Zolgensma (onasemnogene abeparvovec) for the treatment of spinal muscular atrophy (SMA) in children aged 2 years and younger was based on the data of two phase 3 clinical trials: START (NCT02122952) and STR1VE (NCT03306277).
The START study was performed to establish safety and find the appropriate dose of onasemnogene abeparvovec.
The STR1VE study involved 22 children with SMA who were treated after symptoms appeared (symptomatic). All patients received Zolgensma (onasemnogene abeparvovec) through an intravenous infusion over 60 minutes. There was no placebo group. Results were measured against the natural history of SMA.
Results
The primary efficacy outcomes were reaching developmental motor milestones (e.g., head control and the ability to sit without support for at least 30 seconds at the 18-months-of-age study visit) and event-free survival. An event is defined as death, the need for permanent ventilatory support (such as tracheostomy), or the need for respiratory support (not due to illness or surgery) for ≥16 hours a day for at least 14 days.
These outcomes are a co-primary outcome which were assessed in sequence: the measure of functional independent sitting was determined first and, only when this endpoint met statistical significance, was the outcome of event-free survival assessed.
Zolgensma (onasemnogene abeparvovec) significantly increased event-free survival.
- 91% (20/22) of children were alive and did not need permanent breathing support at the 14-months-of-age study visit. These results were maintained through 18 months of age.
A majority of children reached the motor milestone at 16 months of age:
- 59% (13/22) of children could sit without support for ≥30 seconds at 18 months of age
- 64% (14/22) of children could sit without support for ≥30 seconds at any point in the study
- 85% (17/20) of children had head control. At the start of the study, 2/22 patients already had head control before dosing and maintained control
- 59% (13/22) of patients could roll from back to sides
- 1 patient could stand and walk independently. This patient was treated at 1.1 months of age and could stand at 14.7 months and walk at 15.3 months of age.
There is an ongoing long-term follow-up (LTFU) study that monitors the safety of many patients from the START study. The results show the safety and efficacy of Zolgensma up to 5 years after treatment and 5 years of age or older. As of December 2019, 100% (10/10) of patients were alive and did not need permanent breathing support, and 100% (10/10) have maintained motor milestones achieved at the end of the START study.
Please refer to the summary of product characteristics below and in the resource section for comprehensive information about the safety and effectiveness of Zolgensma (onasemnogene abeparvovec) for the approved indication.
References
- Zolgensma - clinical studies Zolgensma.com, cited Mar 2021
- Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Clinical Trial START, cited Mar 2021
- Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 Clinical Trial STR1VE, cited Mar 2021
- Zolgensma STR1VE results Zolgensma-hcp.com, cited Mar 2021
- Full prescribing information [FDA]: Zolgensma (onasemnogene abeparvovec) [PDF] AveXis, May 2019
Price & Costs
Zolgensma (onasemnogene abeparvovec) treatment costs
The cost for a monthly or yearly treatment of Zolgensma (onasemnogene abeparvovec) depends on your prescription requirements which includes the dosage in mg and medicine type (Vial).
The price of the medicines you see on sale is the cost set by the manufacturer. In addition, shipping costs and a Named Patient support fee will apply.
Make an enquiry and we will help you calculate the total price to buy Zolgensma (onasemnogene abeparvovec) online and have it delivered where you live. From your side, we’ll simply need a prescription from your treating doctor. In some countries we might also need an import license. Our Patient Support team will guide you throughout the process.
Zolgensma (onasemnogene abeparvovec) price and additional costs
- The price of Zolgensma (onasemnogene abeparvovec)
- Our Named Patient support fee
- Shipping costs
- Payment methods when you buy Zolgensma (onasemnogene abeparvovec)
The base cost of each medicine is set by the manufacturer. Generally, our Named Patient support fee, shipping costs and any local tax (if applicable) are not included in the product price that is displayed on our website unless stated otherwise.
everyone.org provides access to Zolgensma (onasemnogene abeparvovec) on the Named Patient import basis, which means that we work on behalf of the patient in cooperation with their treating doctor. Our Named Patient support fee covers sourcing, logistics and following up to make sure that the order is received safely and in perfect condition. The Named Patient support fee is tiered relative to the cost of the medicine.
The shipping prices are dependent on the type of shipping necessary for the particular medicine (regular or cold chain) and upon the destination country. The shipping fees may include the shipping from our supplier to our partner pharmacy as well as the shipping costs to deliver the medicine to the patient’s country of residence. The shipping fee might also include the cost of special packaging, such as coolboxes, cool packs and data loggers, as well as transport insurance to guarantee that you receive your medicine in good order.
When you buy Zolgensma (onasemnogene abeparvovec), you can make the payment by bank transfer or by credit card. Payments are fully encrypted and secure. When paying by credit card, you can pay online once you have received your order confirmation. Please be aware that a 2.8% fee applies when paying by credit card.
Zolgensma (onasemnogene abeparvovec) reimbursement
It might be possible for you to claim the cost of Zolgensma (onasemnogene abeparvovec) via your country's healthcare system. Some countries have a government fund providing financial assistance for citizens to access lifesaving medical treatment abroad. The best thing would be to check with your country's own health ministry. We also highly recommend checking out local patient blogs, communities and patient organisations to find out more about your options.
Medical insurance
Generally, we do not support medical insurance, but we do provide you with an invoice after you make the payment that you may send to your medical insurance company so they can reimburse you. In some limited circumstances, we do accept insurance. Contact us for more details. You can pay by bank transfer or credit card.
Crowdfunding
Crowdfunding might also be an option for you. It is a means of raising funds by using an online platform to group together many small donations from friends, family and others, in order to help you afford medical treatment. Here are some crowdfunding platforms where you can seek support: GoFundMe, GiveForward, FundRazr and Watsi.
Shipping
Room temperature shipping
For this medication, standard shipping applies. This means that a consistent room temperature of 15 to 25°C is maintained during transit.
Your order will be shipped to you by our partner couriers, such as DHL, UPS and specialised cold chain couriers. If you would like your order to be shipped by a specific courier, please inform us when you place the order.
Our standard delivery time varies from 5 to 10 working days.
The shipping cost depends on the medicine and the destination country. After making an enquiry, you will receive a detailed quote that includes the shipping costs.
You can also pick up your medicine from our partner pharmacy in Amsterdam or our partner pharmacy in Luxembourg after the order has been paid for.
Our Process
Four steps to access the medicines you need
How and where to access and buy Zolgensma: You can access Zolgensma from everyone.org if the drug has not been approved or is not available in your country.
- Step 1: You send a request for your medicine online
- Step 2: We verify your prescription and medical details
- Step 3: We source your medicine
- Step 4: We deliver your medicine
Submit a request for a specific medicine, or a general request, and your Patient Support Manager will contact you within 24 hours. They will be there to support and guide you in anyway possible to help you get the medicine you need.
Since we help patients access medicines not available in their home country, our first step is to check that the medicine you need is not currently available in your home country. Next we will also verify that you have a prescription from a doctor in your home country for the medicine you need.
Once we have verified your prescription and medical details, our expert sourcing team will work with our global network of suppliers to find you the best price. Once we have found you the lowest possible cost for your medicine, we will present you with a final offer for your approval.
After you have accepted our offer, our experienced logistics team will arrange all the shipping and transportation of your medicine. We will work hard to keep you informed at every step along the way and get your medicine to you quickly and safely. To date we have successfully delivered packages to over 75 countries around the world.
Contact Patient Support
If you have any questions or need any help, contact our Patient Support Team. We will get in touch with you within 24 hours from Monday to Friday between 9:00 and 10:00 CET.
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